Adult Acute Lymphoblastic Leukemia

Treatment (PDQ®)–Patient Version

SECTIONS
 General Information About Adult Acute Lymphoblastic Leukemia
 Stages of Adult Acute Lymphoblastic Leukemia
 Recurrent Adult Acute Lymphoblastic Leukemia
 Treatment Option Overview
 Treatment Options for Adult Acute Lymphoblastic Leukemia
 To Learn More About Adult Acute Lymphoblastic Leukemia
 About This PDQ Summary
 View All Sections

Treatment Option Overview

KEY POINTS

 There are different types of treatment for patients with adult ALL.

 The treatment of adult ALL usually has two phases.

 Four types of standard treatment are used:

o Chemotherapy

o Radiation therapy

o Chemotherapy with stem cell transplant

o Targeted therapy

 New types of treatment are being tested in clinical trials.

o Biologic therapy

o Chimeric antigen receptor (CAR) T-cell therapy

 Patients may want to think about taking part in a clinical trial.

 Patients can enter clinical trials before, during, or after starting their cancer treatment.

 Treatment for adult acute lymphoblastic leukemia may cause side effects.

 Follow-up tests may be needed.

There are different types of treatment for patients with adult ALL.
Different types of treatment are available for patients with adult acute lymphoblastic leukemia (ALL). Some treatments
are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research
study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical
trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment.
Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started
treatment.

The treatment of adult ALL usually has two phases.

The treatment of adult ALL is done in phases:

 Remission induction therapy: This is the first phase of treatment. The goal is to kill the leukemia cells in the blood and bone
marrow. This puts the leukemia into remission.
 Post-remission therapy: This is the second phase of treatment. It begins once the leukemia is in remission. The goal of post-
remission therapy is to kill any remaining leukemia cells that may not be active but could begin to regrow and cause
a relapse. This phase is also called remission continuation therapy.
Treatment called central nervous system (CNS) sanctuary therapy is usually given during each phase of therapy. Because
standard doses of chemotherapy may not reach leukemia cells in the CNS (brain and spinal cord), the cells are able to "find
sanctuary" (hide) in the CNS. Systemic chemotherapy given in high doses, intrathecal chemotherapy, and radiation therapy to the
brain are able to reach leukemia cells in the CNS. They are given to kill the leukemia cells and lessen the chance the leukemia
will recur (come back). CNS sanctuary therapy is also called CNS prophylaxis.

Four types of standard treatment are used:

Chemotherapy

Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping
them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and
can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into
the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavitysuch as the abdomen, the drugs mainly affect cancer
cells in those areas (regional chemotherapy). Combination chemotherapy is treatment using more than one anticancer drug. The
way the chemotherapy is given depends on the type and stage of the cancer being treated.

Intrathecal chemotherapy may be used to treat adult ALL that has spread, or may spread, to the brain and spinal cord. When used
to lessen the chance leukemia cells will spread to the brain and spinal cord, it is called central nervous system (CNS) sanctuary
therapy or CNS prophylaxis.
ENLARGE
Intrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space
that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way,
shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped
container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube
into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the
CSF in the lower part of the spinal column, after a small area on the lower back is numbed.
See Drugs Approved for Acute Lymphoblastic Leukemia for more information.

Radiation therapy

Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them
from growing. There are two types of radiation therapy:
 External radiation therapy uses a machine outside the body to send radiation toward the cancer.
 Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly
into or near the cancer.
The way the radiation therapy is given depends on the type of cancer. External radiation therapy may be used to treat adult ALL
that has spread, or may spread, to the brain and spinal cord. When used this way, it is called central nervous system (CNS)
sanctuary therapy or CNS prophylaxis. External radiation therapy may also be used as palliative therapy to relieve symptoms and
improve quality of life.

Chemotherapy with stem cell transplant

Chemotherapy is given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer
treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed
from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the
stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore)
the body's blood cells.
See Drugs Approved for Acute Lymphoblastic Leukemia for more information.
ENLARGE

Stem cell transplant. (Step 1): Blood is taken from a vein in the arm of the donor. The patient or another
person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood
is returned to the donor through a vein in the other arm. (Step 2): The patient receives chemotherapy to kill
blood-forming cells. The patient may receive radiation therapy (not shown). (Step 3): The patient receives
stem cells through a catheter placed into a blood vessel in the chest.

Targeted therapy

Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells without
harming normal cells. Monoclonal antibody therapy and tyrosine kinase inhibitor therapy are types of targeted therapy used to
treat adult ALL.
Monoclonal antibody therapy is a cancer treatment that uses antibodies made in the laboratory, from a single type of immune
system cell. These antibodies can identify substances on cancer cells or normal substances that may help cancer cells grow. The
antibodies attach to the substances and kill the cancer cells, block their growth, or keep them from spreading. Monoclonal
antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer
cells. Blinatumomab and inotuzumab ozogamicin are monoclonal antibodies used with stem cell transplant to treat adult ALL.
Tyrosine kinase inhibitor therapy blocks the enzyme, tyrosine kinase, that causes stem cells to develop into more white blood
cells (blasts) than the body needs. Imatinib mesylate(Gleevec), dasatinib, and nilotinib are tyrosine kinase inhibitors used to treat
adult ALL.
See Drugs Approved for Acute Lymphoblastic Leukemia for more information.

New types of treatment are being tested in clinical trials.

This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being
studied. Information about clinical trials is available from the NCI website.

Biologic therapy

Biologic therapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a
laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This type of cancer treatment is also
called biotherapy or immunotherapy.

Chimeric antigen receptor (CAR) T-cell therapy

CAR T-cell therapy is a type of immunotherapy that changes the patient's T cells (a type of immune system cell) so they will
attack certain proteins on the surface of cancer cells. T cells are taken from the patient and special receptors are added to their
surface in the laboratory. The changed cells are called chimeric antigen receptor (CAR) T cells. The CAR T cells are grown in
the laboratory and given to the patient by infusion. The CAR T cells multiply in the patient's blood and attack cancer cells. CAR
T-cell therapy is being studied in the treatment of adult ALL that has recurred (come back).
ENLARGE

CAR T-cell therapy. A type of treatment in which a patient’s T cells (a type of immune cell) are changed in
the laboratory so they will bind to cancer cells and kill them. Blood from a vein in the patient’s arm flows
through a tube to an apheresis machine (not shown), which removes the white blood cells, including the T
cells, and sends the rest of the blood back to the patient. Then, the gene for a special receptor called a
chimeric antigen receptor (CAR) is inserted into the T cells in the laboratory. Millions of the CAR T cells are
grown in the laboratory and then given to the patient by infusion. The CAR T cells are able to bind to an
antigen on the cancer cells and kill them.

Patients may want to think about taking part in a clinical trial.

For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research
process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment.
Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may
receive the standard treatment or be among the first to receive a new treatment.

Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do
not lead to effective new treatments, they often answer important questions and help move research forward.

Patients can enter clinical trials before, during, or after starting their cancer
treatment.

Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose
cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or
reduce the side effects of cancer treatment.
Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on
NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on
the ClinicalTrials.gov website.

Treatment for adult acute lymphoblastic leukemia may cause side effects.

For information about side effects that begin during treatment for cancer, see our Side Effects page.
Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects
of treatment for ALL may include the risk of second cancers (new types of cancer). Regular follow-up exams are very important
for long-term survivors.

Follow-up tests may be needed.

Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be
repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may
be based on the results of these tests.
Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if
your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-
ups.